title: Clinical assessment of Optivate®, a high-purity concentrate of factor VIII with von Willebrand factor, in the management of patients with haemophilia A.
abstract: Factor VIII (FVIII) concentrates have revolutionized the treatment of patients with haemophilia A. Concerns over the transmission of viral infections through these products have been addressed through stringent, donor-screening procedures and robust antiviral manufacturing steps. Bio Products Laboratory has developed a high-purity FVIII product with von Willebrand factor, Optivate(®). Its safety, tolerability and efficacy as prophylaxis and treatment of bleeds have been established in long-term studies. Seventy previously treated patients with severe haemophilia A, with ≥ 20 exposure days, were recruited into two long-term, multicentre, open-label studies. The protocols were virtually identical. Patients received Optivate(®) either prophylactically or on-demand. A mean of 159.0 EDs were experienced over 11,320 infusions. Under both conditions, Optivate(®) was well tolerated. Only 10% of patients experienced a treatment-related adverse event; the most commonly reported were headache (4% of patients) and dizziness (3% of patients). The mean number of bleeds/patient over the 2 year treatment period was 23.5 during prophylactic use and 70.4 during on-demand use. In patients treated prophylactically, clinical responses to breakthrough bleeds were rated by physicians as excellent or good and as very helpful or helpful by patients in 95% of bleeds. Clinical responses for on-demand patients were rated as excellent or good by physicians and helpful or very helpful by the patients for 91% of bleeds. There were no viral transmissions or inhibitors. The studies confirm the clinical efficacy and safety of Optivate(®) in both prophylactic and on-demand management of patients with haemophilia A.
authors.affiliation: Klinika Hematologii, Akademia Medyczna w Lublinie, Lublin, Poland.
authors.forename: A
authors.initials: A
authors.lastname: Dmoszynska
authors.forename: K
authors.initials: K
authors.lastname: Kuliczkowski
authors.forename: A
authors.initials: A
authors.lastname: Hellmann
authors.forename: J
authors.initials: J
authors.lastname: Trelinski
authors.forename: J
authors.initials: J
authors.lastname: Kloczko
authors.forename: T
authors.initials: T
authors.lastname: Baglin
authors.forename: C
authors.initials: C
authors.lastname: Hay
authors.forename: D
authors.initials: D
authors.lastname: O'Shaughnessy
authors.forename: K
authors.initials: K
authors.lastname: Zawilska
authors.forename: M
authors.initials: M
authors.lastname: Makris
authors.forename: R
authors.initials: R
authors.lastname: Shaikh-Zaidi
authors.forename: E
authors.initials: E
authors.lastname: Gascoigne
authors.forename: C
authors.initials: C
authors.lastname: Dash
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D004338:Drug Combinations; D005169:Factor VIII / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy*; D006470:Hemorrhage / Q000188:drug therapy* / Q000517:prevention & control; D006490:Hemostatics / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D008875:Middle Aged; D017063:Outcome Assessment, Health Care; D055815:Young Adult; D014841:von Willebrand Factor / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D004338:Drug Combinations; D006490:Hemostatics; C509423:factor VIII, von Willebrand factor drug combination; D014841:von Willebrand Factor; D005169:Factor VIII